Research on Incentive Mechanism of Repurposing Drugs for Rare Diseases
Xiaoling Wu , Hongmei Yuan
Asian Journal of Social Pharmacy ›› 2025, Vol. 20 ›› Issue (4) : 337 -344.
Objective To improve the system of accelerated review and approval, and to speed up the development and marketing of repurposing drugs for rare diseases in China. Methods The relevant concepts of rare diseases and the definition of drug repurposing were introduced so as to sort out the advantages and disadvantages of the research and development of drug repurposing for rare diseases. Then, the incentive mechanisms of the research and development of drug repurposing for rare diseases in China and abroad were compared. Results and Conclusion Some suggestions on improving the incentive mechanism of repurposing drugs for rare diseases such as policy support for talents introduction, capital investment, and innovation capabilities are proposed. Besides, the government should further improve policies for the research and development of repurposing drugs for rare diseases, which can gradually narrow the gap between the research and innovation of drug repurposing in developed countries, thus benefiting the patients of rare diseases.
rare disease / drug repurposing / incentive mechanism / innovation ability
| [1] |
|
| [2] |
|
| [3] |
|
| [4] |
The General Office of the Central Committee of CPC. Opinions on Deepening the Reform of Review and Approval System and Encouraging the Innovation of Medical Products and Devices[Z]. 2017. |
| [5] |
|
| [6] |
|
| [7] |
Board on Health Sciences Policy, Institute of Medicine. Institute of Medicine. Drug repurposing and repositioning: Workshop summary [A]. Roundtable on Translating Genomic-Based Research for Health[C]. Washington, DC: National Academies Press, 2014. |
| [8] |
|
| [9] |
|
| [10] |
|
| [11] |
|
| [12] |
|
| [13] |
|
| [14] |
FDA. Rare diseases at FDA[EB/OL]. (2022-12-13) [2023-05-28]. https://www.fda.gov/patients/rare-diseasesfda#:-:text=The%20Orphan%20Drug%20Act%20is%20a%20law%20passed,such%20designation%20if%20the%20drug%20meets%20specific%20criteria. |
| [15] |
FDA. Introduction to the office of orphan products development (OOPD)[EB/OL]. (2017-05-15)[2023-05- 28]. https://www.fda.gov/media/128018/download. |
| [16] |
|
| [17] |
|
| [18] |
FDA. FDA search orphan drug designations and approvals[EB/OL]. (1983-01-01)[2023-05-28]. https://www.accessdata.fda.gov/scrips/opdlisting/oopd/index.cfm. |
| [19] |
|
| [20] |
FDA. FDA approves pemigatinib for relapsed or refractory myeloid/lymphoid neoplasms with FGFR1 rearrangement[EB/OL]. (2022-08-29)[2023-05-28]. https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-pemigatinib-relapsed-or-refractorymyeloidlymphoid-neoplasms-fgfr1-rearrangement. |
| [21] |
Leukemia & Lymphoma Society. Polycythemia vera facts. No. 13.[EB/OL]. (2012-06)[2023-05-28]. www.lls.org/content/nationalcontent/resourcecenter/freeeducationmaterials/mpd/pdf/polycythemiavera.pdf. |
| [22] |
|
| [23] |
|
| [24] |
FDA. Project Orbis, a framework for concurrent submission and review of oncology products[EB/OL]. (2023-11-01)[2023-05-28]. https://www.fda.gov/aboutfda/oncology-center-excellence/project-orbis. |
| [25] |
FDA. FDA approves ruxolitinib for chronic graft-versushost disease[EB/OL]. (2021-09-22)[2023-05-28]. https://www.fda.gov/drugs/resources-information-approveddrugs/fda-approves-ruxolitinib-chronic-graft-versushost-disease#:-:text=On%20September%2022%2C%202021%2C%20the%20Food%20and%20Drug,adult%20and%20pediatric%20patients%2012%20years%20and%20older. |
| [26] |
NHC. Notice on Issuing the First BatchofRareDiseasesCatalogue[EB/OL].(2018-06-08)[2023-05-28]. http://www.nhc.gov.cn/yzygj/s7659/201806/393a9a37f39c4b458d6e830f40a4bb99.shtml. |
| [27] |
|
| [28] |
|
| [29] |
|
| [30] |
|
| [31] |
|
/
| 〈 |
|
〉 |